Drugs / Odevixibat
Regulatory milestones approvals, filings & regulatory actions · 6 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed Alagille syndrome 2 milestones | |||||
| Filed | US (FDA) | Bylvay | Alagille syndrome | 2023-01-09 | “A second potential indication for Bylvay in patients with ALGS has been submitted as a supplemental New Drug Application to the U.S.” ipsen.com ↗ |
| Filed | EU (EMA) | Bylvay | Alagille syndrome | — | “odevixibat for the treatment of ALGS has been resubmitted to the EMA under a new brand name, Kayfanda” ipsen.com ↗ “A variation seeking authorization for ALGS was also submitted to the EMA in 2022” ipsen.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansions (2) | US (FDA) | BYLVAY | — | 2023-06-13 – 2025-12-19 | fda.gov ↗ |
| Approved | US (FDA) | BYLVAY | — | 2021-07-20 | fda.gov ↗ |
| Approved | EU (EMA) | Bylvay | — | 2021-07-16 | europa.eu ↗ |
Trials 11
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT03608319 | Jul → Aug 2018 | — | Albireo | Completed | No outcome recorded |
| Phase 1 | NCT03082937 | Jan → Mar 2017 | Alagille syndrome, primary biliary cholangitis, progressive familial intrahepatic cholestasis | Albireo | Completed | No outcome recorded |
| Phase 1 | NCT02963077 | Jul 2013 → May 2014 | Alagille syndrome, primary biliary cholangitis, progressive familial intrahepatic cholestasis | Albireo | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT02360852 | Jan 2015 → Oct 2016 | primary biliary cholangitis | Sahlgrenska University Hospital | Terminated | No outcome recorded |
| Phase 36 trials · 2 met primary · 1 missed | ||||||
| Phase 3 | NCT05426733 | Jul 2022 → Oct 2026 expected | biliary atresia | Albireo, an Ipsen Company | Active not recruiting | No outcome recorded |
| Phase 3 | NCT05035030 | Sep 2021 → Dec 2026 expected | Alagille syndrome | Albireo, an Ipsen Company | Active not recruiting | No outcome recorded |
| Phase 3 | NCT04674761 | Mar 2021 → Sep 2022 | Alagille syndrome | Albireo | Completed | Met primary |
| Phase 3 | NCT04336722 | Jul 2020 → Jun 2026 | biliary atresia | Albireo, an Ipsen Company | Completed | Missed primary |
| Phase 3 | NCT03659916 | Sep 2018 → Feb 2024 | progressive familial intrahepatic cholestasis | Albireo, an Ipsen Company | Completed | No outcome recorded |
| Phase 3 | NCT03566238 | May 2018 → Jul 2020 | progressive familial intrahepatic cholestasis type 1, progressive familial intrahepatic cholestasis type 2 | Albireo | Completed | Met primary |
| Phase not stated1 trial | ||||||
| — | NCT04483531 | — | progressive familial intrahepatic cholestasis | Albireo | Approved for marketing | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT02630875 Comparator | Aug 2015 → Mar 2017 | intrahepatic cholestasis | Albireo | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 10
| Date | Issuer | Release |
|---|---|---|
| 2026-07-24 | Ipsen | Results Ipsen provides update on Phase III BOLD trial in biliary atresia ipsen.com ↗
Ipsen (Euronext: IPN; ADR: IPSEY) announced today that the Phase III BOLD trial evaluating Bylvay versus placebo for patients living with biliary atresia (BA) who have already undergone a Kasai hepatoportoenterostomy (HPE) did not meet the primary endpoint of improvement in native liver survival vs placebo. |
| 2025-09-19 | Ipsen | Regulatory Bylvay® (odevixibat) approved in Japan for rare liver disease PFIC ipsen.com ↗
Ipsen (Euronext: IPN; ADR: IPSEY) announced today that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted regulatory approval for Bylvay® (odevixibat) for the treatment of pruritus associated with progressive familial intrahepatic cholestasis (PFIC). |
| 2024-11-18 | Ipsen | Results Bylvay® (odevixibat) data shows sustained improvement in severe itch and serum bile acid levels in patients with PFIC and ALGS ipsen.com ↗
Sustained efficacy data and improvements in height, weight and sleep measures were observed for patients treated with Bylvay for at least 72 weeks in both rare cholestatic diseases. |
| 2024-09-23 | Ipsen | Regulatory Ipsen’s Kayfanda® (odevixibat) approved in European Union for cholestatic pruritus in Alagille Syndrome, a rare liver disease ipsen.com ↗
Ipsen (Euronext: IPN; ADR: IPSEY) today announced that the European Commission has approved Kayfanda® (odevixibat) under exceptional circumstances for the treatment of cholestatic pruritus in Alagille Syndrome (ALGS) in patients aged 6 months or older. |
| 2024-07-26 | Ipsen | Regulatory Ipsen receives CHMP positive opinions for Iqirvo® (elafibranor) in Primary Biliary Cholangitis and Kayfanda® (odevixibat) in Alagille Syndrome, two rare cholestatic liver diseases ipsen.com ↗
Kayfanda® (odevixibat) has also received a positive opinion from CHMP as a treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older. |
| 2023-10-23 | Ipsen | Regulatory Ipsen updates on E.U. Marketing Authorisation Application for odevixibat in Alagille syndrome ipsen.com ↗
Ipsen (Euronext: IPN: ADR: IPSEY) today announced that the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products (COMP) confirmed its negative opinion recommending not to maintain the orphan designation for Bylvay® (odevixibat) in Alagille syndrome (ALGS). |
| 2023-07-21 | Ipsen | Regulatory Correction: Ipsen provides update on E.U. Marketing Authorization Application for Bylvay® for cholestatic pruritus in patients with Alagille syndrome ipsen.com ↗
“We are pleased with today’s CHMP positive opinion which is supported by the data from our Phase III ASSERT trial,” said Howard Mayer, Executive Vice President and Head of Research and Development for Ipsen. |
| 2023-06-13 | Ipsen | Regulatory U.S. FDA approves Bylvay® for patients living with cholestatic pruritus due to Alagille syndrome ipsen.com ↗
Ipsen (Euronext: IPN: ADR: IPSEY) today announced that the U.S. Food and Drug Administration (FDA) has approved Bylvay® (odevixibat) for the treatment of cholestatic pruritus in patients from 12 months of age with Alagille syndrome (ALGS). |
| 2023-06-12 | Ipsen | Results Ipsen to present new data on clinical outcomes and patient validation tools from growing rare liver disease portfolio at EASL 2023 ipsen.com ↗
In the investigational Bylvay indication for patients with ALGS, new pooled data from the Phase III ASSERT trial and extension studies demonstrate significant improvements in pruritus and sBAs and sleep disturbances. |
| 2023-05-17 | Ipsen | Results Ipsen to present new Bylvay® (odevixibat) data at annual ESPGHAN congress, showcasing commitment to furthering treatment for rare cholestatic liver diseases ipsen.com ↗
In PFIC, longer native liver survival suggests there may be a disease-modifying benefit to this treatment,” said Dr. Howard Mayer, Executive Vice President and Head of Research and Development, Ipsen. |
All press releases naming this drug 10 releases
Evidence & citations 10 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | odevixibat | “A4250 (odevixibat)” NCT03566238 ↗ |
| Known as | A4250 | “A4250 (odevixibat)” NCT03659916 ↗ |
| Known as | AR-H064974 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297588 ↗ |
| Known as | AZD-8294 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297588 ↗ |
| Known as | IBAT Inhibitor A4250 | “IBAT Inhibitor A4250” NCT02360852 ↗ |
| Known as | Odevixibat (as sesquihydrate) | ChEMBL registry synonym — accepted as the source's own label CHEMBL4297588 ↗ |
| Action | Inhibit | “Ileal bile acid transporter inhibitor” CHEMBL4297588 ↗1“IBAT Inhibitor A4250” NCT02360852 ↗ |
| Modality | Small molecule | “A4250 is a small molecule” NCT03566238 ↗ |
| Route | Oral | “1.5 - 3 mg orally” NCT02360852 ↗ |
| Target | SLC10A2 | “Ileal bile acid transporter inhibitor” CHEMBL4297588 ↗ |